Cystic fibrosis: NHLBI Workshop on the Primary Prevention of Chronic Lung Diseases

Ann Am Thorac Soc. 2014 Apr;11 Suppl 3(Suppl 3):S161-8. doi: 10.1513/AnnalsATS.201312-444LD.

Abstract

Cystic fibrosis (CF) is a life-limiting, monogenic disorder characterized by chronic sinopulmonary and gastrointestinal involvement. Progressive pulmonary disease leads to death in the majority of patients. Despite its well-defined molecular basis related to defects in the cystic fibrosis transmembrane conductance regulator anion transport channel, there are large gaps in our understanding of the origin of CF lung disease. Disease has been shown to be present in infancy, and there is mounting evidence that abnormalities begin in utero. Heterogeneity of clinical presentations and severity suggest that many factors involved in lung disease have yet to be fully elucidated. Although new advances in therapeutic treatments have shown promise in delaying disease progression, the prevention of pulmonary disease at its origin (primary prevention) should be a key goal of CF care. The objective of this workshop was to (1) review our understanding of the origins of CF lung disease, (2) determine gaps in the knowledge base that are most significant and most likely to enable prevention of CF lung disease, and (3) prioritize new research questions that will promote pulmonary health in both CF and other childhood lung diseases. The goal of this report is to provide recommendations for future research that will improve our understanding of pulmonary development in health and disease, improve outcome measures and biomarkers for early lung disease, and determine therapeutic targets and strategies to prevent the development of lung disease in children with CF.

Publication types

  • Review

MeSH terms

  • Chronic Disease
  • Congresses as Topic*
  • Cystic Fibrosis / prevention & control*
  • Humans
  • Lung Diseases / prevention & control*
  • National Heart, Lung, and Blood Institute (U.S.)
  • Primary Prevention / methods*
  • United States